Abstract: The present invention relates nucleic acid molecules and concatemers containing spacer sequences useful for the efficient packaging of viral particles so as to minimize the incorporation of contaminant nucleic acids into these vectors, as well as methods of producing such viral particles.

Abstract: The present invention relates to nucleic acid molecules containing spacers and methods of using the same.

Abstract: The invention provides regulatory elements, as well as vectors containing the same that may be used to stimulate transcription of a gene of interest in certain tissue types. The transcription regulatory elements described herein may be operably linked to a transgene, such as acid alpha-glucosidase (GAA), so as to promote expression of the GAA transgene in a cell, such as a muscle cell, liver cell, or neuron. The transcription regulatory elements described herein may be operably linked to a therapeutic transgene and used for the treatment of various disorders, such as lysosomal storage diseases, and particularly Pompe disease.

Abstract: The invention provides compositions and methods for stimulating the expression of the human frataxin gene. The compositions described herein can be used, for instance, to produce genes and RNA equivalents optimized for expression in a particular cell type. The compositions and methods that can be used for treating Frederich ataxia. Using the compositions and methods of the disclosure, a patient (e.g., a mammalian patient, such as a human patient) having Frederich ataxia may be administered a plasmid (e.g., a viral vector) that contains a human frataxin gene (hFXN) or an RNA equivalent thereof.

Abstract: The present invention relates nucleic acid molecules and concatemers containing spacer sequences useful for the efficient packaging of viral particles so as to minimize the incorporation of contaminant nucleic acids into these vectors, as well as methods of producing such viral particles.

Abstract: The present disclosure relates to compositions and methods useful for treating glycogen storage disorders, such as type II glycogen storage disorder, also referred to herein as Pompe disease. Using the compositions and methods of the disclosure, a patient (e.g., a mammalian patient, such as a human patient) having Pompe disease may be administered a viral vector, such as an adeno-associated viral (AAV) vector, that contains a transgene encoding acid alpha-glucosidase.

Abstract: The present invention relates nucleic acid molecules and concatemers containing spacer sequences useful for the efficient packaging of viral particles so as to minimize the incorporation of contaminant nucleic acids into these vectors, as well as methods of producing such viral particles.

Abstract: The present invention relates to nucleic acid molecules containing spacers and methods of using the same.

Abstract: The present invention relates to nucleic acid molecules containing spacers that can be packaged into viral particles and methods of producing them. In a first aspect, the invention features a nucleic acid molecule including a first spacer (SSI); a first inverted terminal repeat (ITR1); a cloning site (CS); a second inverted terminal repeat (ITR2); and a second spacer (SS2), such as a eukaryotic spacer; operably linked to each other in a 5?-to-3? direction as: SS1-ITR1-CS-ITR2-SS2. In an embodiment, the invention features a vector comprising any of the above-described nucleic acid molecules. In another aspect, the invention features a plurality of viral particles including the nucleic acid molecule. The invention further includes a host cell including any of the above-described vectors.

Abstract: The invention provides regulatory elements, as well as vectors containing the same that may be used to stimulate transcription of a gene of interest in certain tissue types. The transcription regulatory elements described herein may be operably linked to a transgene, such as acid alpha-glucosidase (GAA), so as to promote expression of the GAA transgene in a cell, such as a muscle cell, liver cell, or neuron. The transcription regulatory elements described herein may be operably linked to a therapeutic transgene and used for the treatment of various disorders, such as lysosomal storage diseases, and particularly Pompe disease.

Abstract: The present invention relates nucleic acid molecules and concatemers containing spacer sequences useful for the efficient packaging of viral particles so as to minimize the incorporation of contaminant nucleic acids into these vectors, as well as methods of producing such viral particles.

Abstract: The present invention relates to nucleic acid molecules containing spacers that can be packaged into viral particles and methods of producing them. In a first aspect, the invention features a nucleic acid molecule including a first spacer (SSI); a first inverted terminal repeat (ITR1); a cloning site (CS); a second inverted terminal repeat (ITR2); and a second spacer (SS2), such as a eukaryotic spacer; operably linked to each other in a 5?-to-3? direction as: SS1-ITR1-CS-ITR2-SS2. In an embodiment, the invention features a vector comprising any of the above-described nucleic acid molecules. In another aspect, the invention features a plurality of viral particles including the nucleic acid molecule. The invention further includes a host cell including any of the above-described vectors.

Abstract: Novel packaging cell lines useful for generating viral accessory protein independent HIV-derived retroviral vector particles, methods of constructing such packaging cell lines and methods of using the viral accessory protein independent HIV-derived retroviral vector particles are disclosed.